Muscular Dystrophy Therapy

New HDAd vector may provide a novel gene therapy strategy for Duchenne muscular dystrophy

Published in Gene Therapy Weekly, October 27th, 2005

Study results suggest that new HDAd vector will provide a novel gene therapy strategy for Duchenne muscular dystrophy, raising the prospects for gene therapy of other hereditary myopathies.

According to a study from Japan, "The helper-dependent adenovirus (HDAd) vector is less immunogenic and has a larger cloning capacity of up to 37 kb enough to carry the full-length dystrophin cDNA. However, high and long-term expression of dystrophin transduced to mature muscle still remains difficult. One of the main reasons for this is that the expression of the coxsackievirus and adenovirus receptor (CAR) is very low in mature muscle."

"We have constructed two...

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Source: Gene Therapy Weekly (2005-10-27)

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